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A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease

StatusRecruiting
PhasePhase 4
Started2026-03-19
View on ClinicalTrials.gov ↗

Amendment history

2026-04-08
minor
Study Status v4
Primary completion date2030-01-31→2030-02-12
Completion date2030-01-31→2030-02-12
Start dateestimated 2026-03-06→confirmed 2026-03-19
2026-02-06
minor
Study Status v3
Primary completion date2029-08-24→2030-01-31
Completion date2029-08-24→2030-01-31
Start date2025-12-23→2026-03-06
2025-11-10
notable
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v2
Trial statusNot Yet Recruiting→Recruiting
Study sites0→1
2025-10-22
minor
Study Status v1
Start date2025-10-09→2025-12-23
2025-08-19
minor
Original filing
148 trials monitored
See 16 trials at risk
This study is researching a drug called pozelimab (called "study drug"). The main aim of this study is to monitor the safety and tolerability of the study drug. The study is focused on young children 1 to 5 years of age, who have CHAPLE disease. CHAPLE is a very rare hereditary disease that can cause potentially life-threatening symptoms related to the stomach and intestines (gastrointestinal symptoms), and symptoms related to the heart and blood vessels (cardiovascular symptoms). The study is also looking at several other research questions, including: * What side effects may happen from taking the study drug * How much study drug is in the blood at different times * Whether the study drug blocks Complement 5 (C5) in the body * Whether the study drug changes the level of a substance called CH50 measured in the blood * Whether the study drug changes the levels of albumin and other proteins * Whether the body makes antibodies against study drug, which could make the study drug less effective or could lead to side effects
Trial Details
NCT Number NCT07142343
Lead Sponsor Regeneron Pharmaceuticals
Conditions CHAPLE Disease
Enrollment 5 participants
Start Date 2026-03-19
Primary Completion 2030-02-12 (estimated)
Study Completion 2030-02-12 (estimated)
Updated on ClinicalTrials.gov 2026-04-13